Trial participants Jack and Tom Bosanquet
A team led by scientists at UCL, funded by the Medical Research Council (MRC) and AVI BioPharma, have made an important breakthrough in the development of a treatment for Duchenne Muscular Dystrophy (DMD). Together with the MDEX Consortium, chaired by Professor Francesco Muntoni (UCL Institute for Child Health), the group show in a paper published in The Lancet today that a gene-based drug treatment was effective in restoring the dystrophin protein that is missing in sufferers of DMD, in seven out of 19 trial participants. DMD is a devastating and life-limiting condition that affects one in 3,500 male births in the general population, with around 100 cases diagnosed in the UK each year. Three of the participants in the two highest dose cohorts showed dystrophin levels that exceeded 18 per cent of those found in normal muscle cells. There was significant statistical increase across the cohorts. Thirteen per cent of boys with DMD could be treated with this gene specific, exon-skipping therapy, the largest group by a single antisense. Overall scientists say this approach could work for at least 70 per cent of DMD sufferers.
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